POS1376 LONG-TERM EFFICACY AND SAFETY OF CANAKINUMAB IN PATIENTS WITH HIDS (HYPER-IgD SYNDROME) - INTERIM ANALYSIS OF THE RELIANCE REGISTRY
Authors: P. Oommen, T. Kallinich, J. Rech, N. Blank, J. Weber-Arden, J. B. Kuemmerle-Deschner
Journal: Annals of the Rheumatic Diseases (2022)

Background Hyper-IgD syndrome/mevalonate kinase deficiency (HIDS/MKD) is a rare autoinflammatory condition caused by a defect in the gene coding for mevalonate kinase. This periodic fever syndrome is characterized by severe systemic and organ inflammation. Treatment with interleukin-1β inhibitor canakinumab (CAN), approved and applied for treatment of HIDS/MKD patients since 2017 [1], resulted in rapid remission of symptoms and normalization of laboratory parameters in most patients in clinical trials [2] as well as in real-life. Objectives To explore the long-term efficacy and safety of CAN under routine clinical practice conditions in pediatric (age ≥2 years) and adult HIDS/MKD patients. Methods RELIANCE is a prospective, non-interventional, multi-center, observational study based in Germany with a 3-year follow-up period. Patients with clinically confirmed diagnoses of TRAPS, CAPS, FMF or HIDS/MKD who routinely receive CAN are enrolled in order to evaluate efficacy and safety of CAN under standard clinical practice conditions at baseline and at 6-monthly intervals. Results The present interim analysis shows baseline data of 8 HIDS/MKD patients enrolled by December 2021 as well as preliminary 18-month data. Of these patients, 5 (63%) were females and median age at baseline was 8 years (2–39 years). The median duration of prior CAN treatment at baseline was 1.5 years (0–5 years). Standard, low, and high dose CAN treatment was evenly distributed at every interval. Preliminary results indicate stable remission and disease control by physicians´ and patients´ assessment as well as laboratory parameters (Table 1). In total, 4 patients were affected by adverse drug reactions, however, none of these events was classified as serious. Table 1. Baseline characteristics and interim analysis data of patients with HIDS. Baseline (N=8 ) 6 months (N=7 ) 12 months (N=6 ) 18 months (N=4 ) Number (%) of patients in disease remission (physician assessment) 4 (50) 6 (86) 4 (67) 3 (75.0) Physician assessment of disease activity, percentage of absent/mild-moderate/severe rating 37 / 50 / 13 71 / 29 / 0 50 / 33 / 17 50 / 50 / 0 Patient´s assessment of current disease activity; 0–10, median (min; max) 0 (0; 7) 2.0 (0; 7) 0.0 (0; 8) 0.0 (0; 0) Patient´s assessment of current fatigue; 0–10, median (min; max) 2.5 (0; 7) 3.0 (0; 7) 1.0 (0; 4) 1.0 (0; 2) Number (%) of patients without impairment of social life by the disease 3 (50) 4 (67) 4 (80) 1 (33) Number (%) of patients with days absent from work/school during last 6 months 2 (25) 3 (43) 0 (0) 1 (25) CRP, median (mg/dl) 0.2 0.5 0.3 2.1 SAA, median (mg/dl) 0.6 0.7 0.8 0.5 ESR, median (mm/h) 10.0 6.0 8.0 13.0 CRP, c-reactive protein; SAA, serum amyloid A; ESR, erythrocyte sedimentation rate Conclusion Baseline characteristics and preliminary data of HIDS/MKD patients from the RELIANCE study indicate good clinical and laboratory disease control and no unexpected safety concerns at the 18 months interim analysis. References [1]Ilaris, INN-canakinumab (europa.eu) [2]De Benedetti F, et al. Canakinumab for the treatment of autoinflammatory recurrent fever syndromes. N Engl J Med 2018;378:1908–19 Disclosure of Interests Prasad Oommen Grant/research support from: Novartis, Tilmann Kallinich Consultant of: Sobi, Novartis, Roche, Grant/research support from: Novartis, Jürgen Rech Speakers bureau: Abbvie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD; Mylan, Novartis, Roche, Sanofi, Sobi, UCB, Consultant of: Abbvie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD, Mylan, Novartis, Roche, Sanofi, Sobi, UCB, Grant/research support from: Novartis, Sobi, Norbert Blank Consultant of: Novartis, Sobi, Lilly, Pfizer, Abbvie, BMS, MSD, Actelion, UCB, Boehringer-Ingelheim, Roche, Grant/research support from: Novartis, Sobi, Julia Weber-Arden Employee of: Novartis, J. B. Kuemmerle-Deschner Consultant of: Novartis, AbbVie, Sobi, Grant/research support from: Novartis, AbbVie, Sobi